Bayer's Cell Therapy for Replacing Dopamine Neurons in Parkinson’s Advances to Phase III
Bayer, through its subsidiary BlueRock Therapeutics, has announced plans to advance bemdaneprocel (BRT-DA01), its investigational pluripotent stem cell-derived dopaminergic neuron therapy for Parkinson’s disease, into a registrational Phase III clinical trial. The trial, named exPDite-2, will be the first Phase III study of an allogeneic pluripotent stem cell-derived therapy for the neurodegenerative condition. Enrollment of approximately 102 participants is expected to begin in the first half of 2025.
Bemdaneprocel is engineered to replace dopamine-producing neurons lost in Parkinson’s disease. Derived from pluripotent stem cells, the therapy is implanted directly into the brain as dopaminergic neuron precursors. Once implanted, these precursors develop into mature neurons capable of potentially restoring neural networks and addressing both motor and non-motor symptoms of the disease.
The trial, a sham-surgery controlled, double-blind study, will assess bemdaneprocel’s efficacy and safety in people with moderate Parkinson’s disease. Its primary endpoint will measure changes in ON-time without troublesome dyskinesia from baseline to week 78 (the trial will evaluate how much time patients experience effective symptom relief without being disrupted by severe dyskinesia over the specified period). Secondary endpoints will evaluate motor function, safety, tolerability, daily living activities, and quality of life. Findings from the study may contribute to a regulatory submission for potential approval.
Data from a Phase I trial, which enrolled 12 participants, showed that bemdaneprocel was well-tolerated, with no serious adverse events related to the therapy reported 24 months post-surgery. The study also showed trends of motor function improvement. These participants remain under long-term evaluation.
According to Bayer, the Phase III trial builds on the regenerative medicine advanced therapy (RMAT) designation granted to bemdaneprocel by the FDA in 2024 and is a significant milestone in its cell and gene therapy pipeline. BlueRock Therapeutics, the subsidiary responsible for developing bemdaneprocel, will continue leading the investigational therapy’s clinical program.
BlueRock Therapeutics' pipeline spans multiple therapeutic areas—neurology, cardiology, ophthalmology and immunology:
- Parkinson’s Disease (bemdaneprocel, BRT-DA01); Dopaminergic neuron therapy for Parkinson’s disease (Phase III, planned initiation in H1 2025).
- Parkinson’s Disease (BR-DA02); Dopaminergic neuron therapy for Parkinson’s disease (Preclinical).
- Neurodegenerative Disease; Microglia-based therapy addressing neurodegenerative conditions (Preclinical).
- Genetic or Acquired Myelin Defects; Oligodendrocyte-based therapy for conditions involving myelin damage (Preclinical).
- Primary Photoreceptor Diseases (OpCT-001); Photoreceptor cell therapy in partnership with Opsis Therapeutics (Phase I).
- Early/Intermediate Dry AMD; Retinal pigment epithelium (RPE) cell therapy for age-related macular degeneration (Preclinical).
- Late Dry AMD / Geographic Atrophy; Combined photoreceptor and RPE therapy for advanced dry AMD (Preclinical).
- Heart Failure (BR-CM01); Cardiomyocyte-based therapy for heart failure (Preclinical).
- Autoimmune Disease; CD4+ T-Reg cell therapy in partnership with bit.bio (Preclinical).
Bayer acquired full ownership of BlueRock Therapeutics in August 2019. The acquisition valued BlueRock at approximately $1 billion, including Bayer's initial 40.8% stake. BlueRock continues to operate as an independent entity under Bayer, maintaining its focus on developing regenerative therapies using its CELL+GENE platform.
Topics: Novel Therapeutics
